Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
How would you define the primary site GTV and CTV volumes in a high risk neuroblastoma patient who obtained a complete response to induction chemo?
This scenario is exceedingly rare. My opinion would probably depend on staging/histology/MYCN status. Some of the historic HR NBL cases are now being shifted into Intermediate Risk and there have been favorable outcomes without the routine use of RT. So if it was a Stage 3 365-547d, MYCN-NA, UH, any...
Is there any benefit to using desmopressin over vWF replacement therapy for vWD?
For mild type 1 and 2A, we do offer DDAVP testing and use. It was a much more useful drug when it was widely available as a nasal spray. It still has its place, but with patients whose level of VWF is <20% baseline, we have a lot of treatment failures and end up using factor.
Do you recommend menstrual suppression for post-menarchal adolescents and young adults receiving myelosuppressive chemotherapy?
One other point of consideration is the potential benefit of GnRH agonists (i.e., Lupron) on the reproductive potential of post-pubertal females given the risk of chemotherapy in decreasing years of fertility (i.e., earlier menopause). Though data on GnRH agonists is limited to females with breast c...
How do you approach isolated CNS recurrence in previously treated neuroblastoma?
As with other pediatric cancers, the CNS can be a sanctuary site for relapse given the poor CNS penetration of many conventional therapeutics used in the front line setting. As such, there has been an increasing frequency of CNS failures which can occur as early as a year to as late as 5-10 years in...
Do you recommend routine use of Evusheld for pre-exposure prophylaxis for patients on immunosuppression?
The use of monoclonal antibodies as passive immunity for pre-exposure prophylaxis is an exciting development for vulnerable patients, including immunosuppressed patients (either primary or through medications such as for autoimmune diseases), cancer patients, and organ transplant recipients. Evushel...
How would you treat a solitary intracranial oligometastasis from Ewing's sarcoma that has been resected?
While an uncommon occurrence in pediatric sarcomas, as much as 2-3% of pediatric RMS and EWS cases can metastasize to the CNS. The optimal management strategy has not been evaluated systematically in prior trials due to the rarity of the event. It’s worth noting that many patients with oligometast...
Do you use FVIII levels to differentiate between DIC and coagulopathy of liver disease?
DIC is a clinical diagnosis that is difficult to establish in the absence of bleeding or thrombosis, particularly in patients with liver disease. I do think that following DIC laboratory markers (FDP, fibrinogen, D-dimer) serially may be helpful as you would not expect them to acutely drop simply be...
What approach do you use when treating adult diffuse gliomas with H3-G34 mutations, now classified into their own category under the 2021 glioma guidelines?
Although DHG H3G34-mutant diffuse gliomas are more commonly associated with pediatric patients, they can occur in young adults as well. In a recent paper describing 17 patients harboring this rare mutation, the median age at diagnosis was 25 years (range: 19–33). All tumors were hemispheric. All cas...
How do you counsel patients with elevated factor VIII levels for their thrombosis risk?
I don't check the factor VIII level as part of a thrombosis workup because the result rarely if ever affects my management of these patients. I would explain to the patient that factor VIII levels are a function of several variables, including age, vascular health (and hence blood pressure, smoking,...
How do you decide between HSCT and immunosuppressive therapy for treating hepatitis associated aplastic anemia?
For young people with a donor, the primary therapy is transplantation. It is curative in the vast majority of patients, hepatitis-associated AA is much less likely to respond to immunotherapy.